The field of the invention relates to methods for inducing polyploidization of megakaryocytes and for treating blood and bone marrow diseases and disorders including leukemias. The field of the invention also relates to methods for identifying agents for treating blood and bone marrow diseases and disorders including leukemias.
Leukemia is a disease that develops when blood cell precursors grow excessively. There are many different forms of leukemia, which are classified by the type of blood cell that is affected or by the genetic abnormality that causes the disease. Unfortunately, there is no one treatment that works for all types, and in general, the most effective therapies are specifically targeted to the molecular causes of each disease. One very aggressive form of blood cancer for which there are few promising treatment options is Acute Megakaryocytic Leukemia (AMKL). This malignancy, which is generally fatal within one year of diagnosis, is caused by the uncontrolled growth of megakaryocytes, the cells that release platelets into the bloodstream to control bleeding. New treatments for leukemias such as AMKL are desirable.